A Study About Gene Therapy for Early Alzheimer’s Disease (AD) and Mild Cognitive Impairment (MCI)

Official Title
A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of AAV2-BDNF [Adeno-Associated Virus (AAV)-based, Vector-Mediated Delivery of Human Brain Derived Neurotrophic Factor] in Subjects with Early Alzheimer’s Disease and Mild Cognitive Impairment

The primary aim of this study is to determine whether administration of the nervous system growth factor "Brain-Deruved Neurotrophic Factor" (BDNF) to eligible patients with Alzheimer's disease and Mild Cognitive Impairment is safe and tolerable. This study will be used to determine whether BDNF administration reduces neuronal loss and rebuilds synapses in the brain of patients with Alzheimer's Disease (AD) and Mild Cognitive Impairment (MCI). 

Could this study be right for you?
  • Male and Females aged 50 years or older
  • Diagnosis of dementia due to Alzheimer's Disease/Diagnosis of Mild Cognitive Impairment (MCI) due to Alzheimer’s Disease
  • A caregiver is available who has frequent contact with the subject, agrees to observe for adverse events, and will accompany the subject to all clinic visits for the duration of the protocol
  • CT or MRI scans within 24 months prior to screening without evidence of an infection, infarction, or other focal 

Inclusion Criteria varies, please contact Study Coordinator for more information.

The below Exclusion Criteria apply to both the AD and MCI groups.

  • Any significant neurological disease other than suspected incipient disease
  • History of schizophrenia 
  • History of systemic cancer within the past 18 months (non-metastatic skin cancers are acceptable).
  • Any significant systemic illness or unstable medical conditions which could lead to difficulty complying with the protocol
  • Use of any investigational drugs within thirty days or five half-lives, whichever is longer, prior to screening.

Exclusion Criteria varies, please contact Study Coordinator for more information. 

Age Range
50 and up